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1·Is it time to revisit orphan drug policies?

是重新审视孤儿药政策的时候吗？

2·Objective: To introduce American Orphan Drug System.

目的：介绍美国罕见病药物制度。

3·Sabril was designated as an orphan drug by the FDA for use in treating infantile spasms.

喜保宁被FDA指定为用于治疗婴儿痉挛的稀少药品。

4·To obtain orphan drug designation, drugs must be for the treatment, prevention or diagnosis of a rare disease or condition.

孤儿药物审批流程需要该药物可以用于治疗、预防或者诊断罕见疾病。

5·Orphan drug status provides the company with financial incentives to promote the development of a drug to treat a rare disease or condition.

稀少药品状态提供给药品公司金融鼓励，促使药品公司开发治疗稀少疾病的药物。

6·Orphan drugs are either drug or biologic products used to treat diseases or conditions affecting fewer than 200,000 people in the United States.

孤儿药物包括药物或者生物制品，用于治疗疾病，并且在美国发病人数少于20万人。

7·It is our intention to find ways to continue the development of our GVAX Pancreas Vaccine using the benefits conferred by gaining Orphan Drug designation.

Simes说，“我们的目的是利用获得指定的孤儿药物权带来的益处，继续GVAX的研发。”

8·Orphan drug designation is available for drugs already approved for another disease or condition or for experimental drugs that have not been previously approved.

已经被批准用于其它疾病治疗的药物或者是尚处于试验阶段未被批准的药物都可以通过孤儿药物申报程序进行。

9·Methods: The historical background formed American Orphan Drug system, regulation of Orphan Drug system, preferential measure, and FDA s approval procedure are stated.

方法：根据个人掌握的资料，说明美国罕见病药物制度形成的背景、有关法规、优惠措施及FDA审批程序。

10·ABSTRACT: OBJECTIVE: to introduce high-value drug insurance access mechanism in Australia, and to provide suggestion for improving orphan drug medical insurance access in China.

摘要：目的：介绍澳大利亚高值药物医保准入机制，为我国罕用药医保准入的完善提供建议。